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  2. Pfizer's gene therapy cuts hemophilia A bleeding rate in late ...

    www.aol.com/news/pfizers-hemophilia-gene-therapy...

    If approved, Pfizer's hemophilia A gene therapy will compete with BioMarin Pharmaceutical's one-time treatment Roctavian, which was approved in the U.S. last year and is priced at $2.9 million.

  3. US FDA approves BioMarin's gene therapy for hemophilia A - AOL

    www.aol.com/news/us-fda-approves-biomarins-gene...

    Roctavian is the first gene replacement therapy for the most common form of hemophilia, enabling patients a way to forego or reduce the need for lifetime treatment with factor proteins needed to ...

  4. FDA approves Pfizer's first gene therapy for rare inherited ...

    www.aol.com/news/fda-approves-pfizer-first-gene...

    The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...

  5. Giroctocogene fitelparvovec - Wikipedia

    en.wikipedia.org/wiki/Giroctocogene_fitelparvovec

    Giroctocogene fitelparvovec (PF-07055480) is an experimental gene therapy for hemophilia A via a recombinant adeno-associated virus serotype 6-based vector. [1]

  6. Etranacogene dezaparvovec - Wikipedia

    en.wikipedia.org/wiki/Etranacogene_dezaparvovec

    Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. [5] [6] [7] Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. [7]

  7. $2.9 million gene therapy for severe hemophilia is approved ...

    www.aol.com/news/gene-therapy-severe-hemophilia...

    The price is less than the $3.5 million announced last year for a similar gene therapy for hemophilia B, a less common form of the disease. Like most medicines in the U.S., the new treatment will ...

  8. Spark Therapeutics - Wikipedia

    en.wikipedia.org/wiki/Spark_Therapeutics

    Fidanacogene elaparvovec, previously known by its study ID number SPK-9001, [12] is a gene therapy for the treatment of hemophilia B. It was developed by Spark in partnership with Pfizer . Fidanacogene elaparvovec is an adeno-associated viral vector which is designed to transfer a working copy of the Factor IX gene into the livers of patients ...

  9. FDA approves CSL gene therapy for hemophilia - AOL

    www.aol.com/news/fda-approves-csls-gene-therapy...

    The companies expect the gene therapy to serve as a one-time shot to cut the rate of annual bleeding for hemophilia B patients, who represent about 15% of all patients with hemophilia. ...

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